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Targeting nuclear RNA for in vivo correction of myotonic dystrophy

Authors :
Wheeler, Thurman M.
Leger, Andrew J.
Pandey, Sanjay K.
MacLeod, A. Robert
Nakamori, Masayuki
Cheng, Seng H.
Wentworth, Bruce M.
Bennett, C. Frank
Thornton, Charles A.
Source :
Nature. August 2, 2012, Vol. 488 Issue 7409, p111, 7 p.
Publication Year :
2012

Abstract

Antisense oligonucleotides (ASOs) hold promise for gene-specific knockdown in diseases that involve RNA or protein gain-offunction effects. In the hereditary degenerative disease myotonic dystrophy type 1 (DM1), transcripts from the [...]

Subjects

Subjects :
RNA -- Physiological aspects -- Health aspects
Myotonic dystrophy -- Genetic aspects -- Physiological aspects -- Development and progression
Oligonucleotides -- Physiological aspects -- Health aspects -- Genetic aspects
Environmental issues
Science and technology
Zoology and wildlife conservation

Details

Language :
English
ISSN :
00280836
Volume :
488
Issue :
7409
Database :
Gale General OneFile
Journal :
Nature
Publication Type :
Academic Journal
Accession number :
edsgcl.299988795

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